关于报告
Rare diseases have increasingly been discussed in the media over the past several years as the number of new treatments has surged. Many of these diseases are life-threatening or life-limiting, and while some conditions can now be treated and managed as chronic conditions, for the first-time new curative treatments are bringing the prospect of a better life to patients. This report updates an analysis of the Orphan Drug market published by the Institute in October 2017 and provides a historical perspective on the characteristics of rare diseases, their treatments and the role of the Orphan Drug Act of 1983 in advancing rare disease medicines. It describes the characteristics of orphan drug spending, volumes and prices, placing orphan drugs in the context of specialty drug trends and overall medicine spending levels and growth.
Report Summary
Many rare diseases remain without effective treatments despite over 500 orphan drug therapies having been approved in the United States. However, a growing focus by researchers and the FDA on rare disease therapies have resulted in a dramatic increase in the number of new therapeutic options for these patients over the past two years. In 2017, the FDA approved 80 orphan indications and in 2018, it approved 57 in the first eight months of the year. The combination of scientific advances, along with a growing commitment by policy makers to advance precision medicine, is fueling the increased number of orphan therapies.
对于患者和付款人来说，孤儿药可能很昂贵。但是，考虑到孤儿药是针对小型患者人群开发的，这些疗法的价格与其使用量之间通常存在反比关系。尽管2017年孤儿药的年中位成本超过46,800美元，但最多的患者使用的前十种稀有疾病疗法的年中位数中位数却低得多，为1,216美元。总体而言，美国的药物支出是由于强调慢性疾病的大量低成本药物的重视，这些药物涉及体积较低但在患者结局方面具有较高价值的药物。
The orphan drug share of the total volume of pharmaceuticals used in the United States declined from a peak of 0.6% in 2003 to 0.3% in 2015 but has risen to 0.4% by 2017. Total drug spending in the United States in 2017 was $451 billion, with almost 56% spent on non-orphan traditional drugs, 34.7% spent on non-orphan specialty drugs and 9.6% spent on orphan indications of approved orphan drugs. Spending on orphan indications has increased moderately and these drugs represent only a small part of the overall medicine budget.
Key Findings
Since the passage of the Orphan Drug Act, the number of orphan indications approved in the United States has risen dramatically
- FDA在2017年的前八个月内批准了80个新的孤儿迹象和57次新孤儿，这是《孤儿毒品法》以来每年最多的数字。
- The FDA granted orphan designations to over 429 unique drugs under development in 2017.
- Drug manufacturers continue to increase their focus on the development of therapies for orphan indications and half of the 42 new active substances (molecules not previously approved as a medicinal product) launched in the United States in 2017 were orphan drugs.
用于治疗小种群的孤儿药通常比用于较大种群的孤儿药物贵
- 治疗的每年成本与患者数量之间存在反比关系，而较昂贵的疗法被分配给相对较少的患者。
- The median annual cost for an orphan drug in 2017 was over $46,800 per year.
- 但是，最多的患者使用的十种孤儿疗法平均每年为9,676美元（中位成本为1,216美元）。
Medicine spending in the United States is shifting from traditional small molecule drugs towards specialty medicines that treat relatively few people with chronic, complex or rare diseases
- 特殊份额的医学支出总额已从1997年的11％上升到2017年的43％，而同期使用孤儿药的支出已从4％上升到10％。
- Many orphan drugs are specialty products, and 87% of orphan spending falls within specialty.
- 毒品支出的演变伴随着医疗保健利益相关者引入的节省成本机制，包括：健康计划专业级别，患者共同保险费，使用专业药房和有限的分销网络等，这些层次增加了患者的成本负担必威手机APP。
尽管美国所有药品支出的四分之一都与孤儿批准的毒品相关
- 虽然被批准的孤儿药物数量从2013年的364种增加到2017年的487，但与孤儿指示使用相关的药物支出的份额从8.1％增加到9.6％，高于1993年的3％。
- 这些分子的非孔量使用占整体药物支出的15.3％，这反映出对于那些具有孤儿和非孔指示的分子，非孔子适应症代表了其大部分的使用和销售。
- 使用孤儿毒品的批准孤儿指示占约430亿美元。
